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From a few days ago: "Prosensa announces 12 presentations at the 19th International Congress of the World Muscle Society in Berlin"Wonder if SRPT will issue a PR on what they are presenting as well --- or perhaps focus more on why they keep pushing out their own timelines --- years of timelines pushed forward actually. Yes, there will be those who blame others for those miscellaneous missed dates - but they begin to add up in years, not months. From Prosensa's press release: LEIDEN, The Netherlands, Sept. 9, 2014 (GLOBE NEWSWIRE) -- Prosensa Holding N.V. (NASDAQ: RNA), the biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today announced that it will be presenting 12 abstracts at the 19th International Congress of the World Muscle Society (WMS), to be held in Berlin, Germany from October 7-11, 2014. Dr. Giles Campion, Prosensa's Chief Medical Officer and co-author of several presentations, said "The wide range and high quality data reported across twelve presentations at WMS, including biomarkers to measure disease progression, clinical results, and our pre-clinical work on multi-exon skipping, which could address the rarer mutations in the dystrophin gene, highlights the scientific rigor and pioneering spirit of our outstanding scientists and collaborators. We will continue to work together to deliver much needed therapies as quickly and efficiently for boys with DMD." A schedule highlighting Prosensa's abstracts at WMS 2014 follows: Oral Presentations In the session "Muscular Dystrophies" on Thursday 9 October from 11:00: •G.O.10 "Skeletal muscle, cardiac, and pulmonary imaging biomarkers of disease activity in boys with Duchenne muscular dystrophy" Authors: A. Mankodi; R. Janiczek; M. Froeling; N. Azzabou; L. Gaur; D. Stock; R. Evers; C. Bishop; L. Yao; C. Grunseich; A. Arai; P. Carlier •G.O.11 "Longitudinal quantitative muscle MRI in 5 Duchenne boys treated with exon 51 skipping - a pilot study" Authors: M.T. Hooijmans; B.H.A.Wokke; N. Goemans; G. Campion; J.G.M. Verschuuren; EH. Niks; H.E. Kan In the session "Treatment approaches in the clinic" on Saturday 11 October from 9:00: •G.O.23 "Drisapersen: An overview of the clinical programme to date in Duchenne Muscular Dystrophy (DMD)" Authors:CM. McDonald; E. Mercuri; N. Goemans; T. Voit; R. Wilson; C. Wardell; G. Campion Poster Presentations In the session "DMD 1 experimental and animal approaches" on Wednesday 8 October from 14:30: •G.P.86 "Digital droplet RT-PCR for the absolute quantification of exon skipping induced by antisense oligonucleotides in (pre-)clinical development for Duchenne muscular dystrophy" Authors: I.G.M. Kolfschoten; R.C. Verheu; J.C.T. van Deutekom; N.A. Datson •G.P.87 "Prospects for single antisense oligonucleotide-induced multiple exon skipping for rare non-hotspot mutations in Duchenne muscular dystrophy" Authors: J.A.M. Janson; I.G.M. Kolfschoten; R.E.Y. van den Eijnde; R. Weij; R.C. Verheul; A. Baghat; M.M. Plug; P.C. de visser; J.C.T. van Deutekom In the session 'DMD 2 Therapeutic evaluations and approaches' on Wednesday 8 October from 16:00: •G.P.107 "Complementary human skin models as a tool to study oligonucleotide-induced injection site reactions" Authors: C. den Besten; T. Steevels; P. Ekhart; S. Jones; S. Gibbs; G. Campion •G.P.108 "Transient proteinuria with oligonucleotide therapy due to interference with tubular protein reabsorption" Authors: G. Campion; C. den Besten; S. Jones; M. Wilme; R. Masereeuw •G.P.113 "Drisapersen (DRIS) treatment for Duchenne muscular dystrophy (DMD): results of up to 188 weeks' follow-up of an open-label extension study" Authors: N. Goemans; M. Tulinius, R. Wilson; C. Wardell; P Bedwell; G. Campion •G.P.114 "Evaluation of efficacy and safety baseline parameters in patients with Duchenne muscular dystrophy (DMD) from three placebo-controlled studies of drisapersen (DRIS)" Authors: E. Mercuri; T. Voit; N. Goemans; CM. McDonald; R. Wilson; C. Wardel; G. Campion •G.P.115 "Pooled analyses of efficacy parameters in patients with Duchenne muscular dystrophy (DMD): results from the drisapersen (DRIS) clinical trial" Authors: N. Goemans; M. Tulinius, R. Wilson; C. Wardell; P Bedwell; G. Campion •G.P.116 "SCOPE - DMD, an EU FP7 funded consortium for skipping trial across Europe in Duchenne muscular dystrophy" Authors: O. Veldhuizen; G. Campion; A. Morgan; H. Aygun; S. Wojczewski; T. Voit; P. Carlie; J. Verschuuren; A. Aartsma - Rus; V. Straub In the session 'DM1 + Myasthenia + Channel diseases' on Wednesday 8 October from 16:00: •G.P.132 "Pre-clinical development of peptide-conjugated antisense oligonucleotides for myotonic dystrophy type 1 (DM1)" Authors: J.C.T. van Deutekom; S.A.M. Mulders; B. Aguilera; A. Gonzalez-Barriga; J. van de Giesse; W.J.A. van den Broe; D.G. Wansink; N.A. Datson |
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Msg # | Subject | Author | Recs | Date Posted |
10394 | Re: From a few days ago: "Prosensa announces 12 presentations at the 19th International Congress of the World Muscle Society in Berlin" | starfe1 | 1 | 9/12/2014 11:47:20 AM |